Gene Therapy for ACM Due to a PKP2 Pathogenic Variant | NCT06109181

Studies
Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

NCT06109181

Sponsor

Lexeo Therapeutics

Status

Active, not recruiting

Last Update Posted

Nov 12, 2025Actual

Enrollment

10Estimated

Phase

Phase 1Phase 2

Conditions

Arrhythmogenic Cardiomyopathy
PKP2-ACM
PKP2-ARVC

Interventions

LX2020

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2025

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Feb 29, 2024Actual

Primary Completion Date

Dec 2026Estimated

Completion Date

Mar 2027Estimated

First Submitted Date

Oct 25, 2023

First Submission Date that Met QC Criteria

Oct 25, 2023

First Posted Date

Oct 31, 2023Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 7, 2025

Last Update Posted Date

Nov 12, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM

Detailed Description

Not provided

Conditions Module

Conditions

Arrhythmogenic Cardiomyopathy
PKP2-ACM
PKP2-ARVC

Keywords

Arrhythmogenic Cardiomyopathy
ACM
Cardiomyopathy
ARVC
Arrhythmogenic Right Ventricular
Arrhythmogenic Right Ventricular Dysplasia
Genetic cardiomyopathy
Gene Therapy
PKP2 Gene
Plakophilin-2
LX2020
HEROIC-PKP2
Ventricular Arrhythmia
PVCs
Sudden Cardiac Death
Cardiac Arrest

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

10Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
LX2020	Experimental	Single ascending dose of LX2020, with a starting dose of 2.0 x10^13 gc/kg, in multiple cohorts	Genetic: LX2020

Interventions

Name	Type	Description	Arm Group Labels	Other Names
LX2020	Genetic	LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)	LX2020

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).	Evaluation of Safety and Tolerability of LX2020	12 months

Secondary Outcomes

Not provided

Other Outcomes

Measure	Description	Time Frame
Selected Exploratory	Change in the frequency of ventricular arrhythmias from baseline.	12 months

Eligibility Module

Eligibility Criteria

Selected Inclusion Criteria:

Adults with a clinical diagnosis of ACM meeting the 2010 revised Task Force Criteria (TFC)
Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2
Frequent premature ventricular complexes (PVCs)
Implantable cardioverter-defibrillator (ICD) implantation ≥ 12 weeks prior to the pre-screening MRI
Left ventricular ejection fraction ≥ 40%

Selected Exclusion Criteria:

Evidence of variant(s) in addition to PKP2 that meets the standard criteria to be considered pathogenic or likely pathogenic for ACM
Other cardiac abnormalities as specified in the protocol
New York Heart Association Functional Class IV at the time of consent
History of prior gene transfer therapy

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

65 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
LEXEO Clinical Trials	Lexeo Therapeutics	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Stanford University		Stanford	California	94305	United States
Johns Hopkins University

Condition Browse Module

MeSH Terms

ID	Term
D009202	Cardiomyopathies
D019571	Arrhythmogenic Right Ventricular Dysplasia
D016757	Death, Sudden, Cardiac
D006323	Heart Arrest

Ancestor Terms

ID	Term
D006331	Heart Diseases
D002318	Cardiovascular Diseases
D006330	Heart Defects, Congenital
D018376	Cardiovascular Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided